Our initial focus is on supporting drug repurposing research using animal models of MEPAN. Repurposing projects test thousands of FDA-approved drugs to identify existing medicines that may address MEPAN symptoms.
Because these drugs have already been reviewed by the FDA and tested in adult patients for safety, repurposing is potentially the fastest, most cost-effective path to finding a treatment.
A collaborative effort between the University of San Francisco, Perlara, Baylor University, Sheba Medical Center in Israel, and the University of Oulu in Finland has identified several drugs that may help patients. Read a blog post with more, or learn about other projects underway or being considered here.
Create models of MECR gene mutations in yeast
Screen 1,000s of existing FDA-approved drugs
Submit to FDA for clinical trial
Test promising drug candidates in patient skin cells